Prosensa and GlaxoSmithKline initiate development of four additional products under existing alliance in Duchenne Muscular Dystrophy. - PPMD Community2020-06-07T09:10:51Zhttp://community.parentprojectmd.org/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A86006&feed=yes&xn_auth=noKaterina,
Please see the int…tag:community.parentprojectmd.org,2010-07-08:1187424:Comment:875732010-07-08T13:48:35.528ZMelanie Sunnyhttp://community.parentprojectmd.org/profile/FengFeng
Katerina,<br />
<br />
Please see the internet addresses below.<br />
<br />
<b>Dr. Giulio Cossu's Home Address:</b><br />
<a href="http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/giulio-cossu/" target="_blank">http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/giulio-cossu/</a><br />
<br />
<b>Stem Cell and Muscular Dystrophies Web Page:…</b>
Katerina,<br />
<br />
Please see the internet addresses below.<br />
<br />
<b>Dr. Giulio Cossu's Home Address:</b><br />
<a href="http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/giulio-cossu/" target="_blank">http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/giulio-cossu/</a><br />
<br />
<b>Stem Cell and Muscular Dystrophies Web Page:</b><br />
<a href="http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/" target="_blank">http://www.scienceparkrome.eu/research/stem-cells-and-muscular-distrophies/</a><br />
<br />
On Stem Cell and Musuclar Dystrophies page, it mentions "<i>Based on these discoveries a phase I/II clinical trial is planned for Duchenne Muscular Dystrophy</i>.". I emailed and asked him when a phase I/II clinical trial will be able to start for Duchenne Muscular Dystrophy, and he replied to me with above email.<br />
<br />
I do believe this method will suit all DMD boys no matter what mutations they are carrying.<br />
<br />
I don't understand the <b>logistic</b> problem for the trial. This trial is extremely costly, and I guess this is the economic problem. As I know <b>CureDuchenne</b> has provided a one year grant for this research.<br />
<br />
Best,<br />
Melanie As such, this will work for D…tag:community.parentprojectmd.org,2010-07-08:1187424:Comment:875672010-07-08T13:35:19.153ZRAKTIM SINGHhttp://community.parentprojectmd.org/profile/RAKTIMSINGH
As such, this will work for DMD kids but one of the important criteria here is 'FULLY HLA-matched donor (a brother or a non carrier sister).' I think that will be tough to find.
As such, this will work for DMD kids but one of the important criteria here is 'FULLY HLA-matched donor (a brother or a non carrier sister).' I think that will be tough to find. Thank you for this news. I ca…tag:community.parentprojectmd.org,2010-07-08:1187424:Comment:875642010-07-08T13:15:18.326ZKaterinahttp://community.parentprojectmd.org/profile/Katerina
Thank you for this news. I can't find any information about this trial and method in the internet, could you me the internet address? thank you in advance. This methos suits to all mutations?<br />
And I don't understand what is the main goal of this trial - to prove it safety or to reach therapeutic effect? They write "the number of patients that we will be able to treat will be small because of logistic and economic problems" - why? if this method will work - doesn't it mean that they help all…
Thank you for this news. I can't find any information about this trial and method in the internet, could you me the internet address? thank you in advance. This methos suits to all mutations?<br />
And I don't understand what is the main goal of this trial - to prove it safety or to reach therapeutic effect? They write "the number of patients that we will be able to treat will be small because of logistic and economic problems" - why? if this method will work - doesn't it mean that they help all boys, why not? Good news about the stem cell…tag:community.parentprojectmd.org,2010-07-08:1187424:Comment:875302010-07-08T00:31:36.738ZOfelia Marinhttp://community.parentprojectmd.org/profile/OfeliaMarin
Good news about the stem cell progressing I think! Thanks for sharing!
Good news about the stem cell progressing I think! Thanks for sharing! MAY GOD BLESS.
cheryl cliff…tag:community.parentprojectmd.org,2010-07-07:1187424:Comment:874392010-07-07T15:11:59.739ZMelanie Sunnyhttp://community.parentprojectmd.org/profile/FengFeng
MAY GOD BLESS.<br />
<br />
<cite>cheryl cliff said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?id=1187424%3ATopic%3A85920&amp;page=2#1187424Comment87432"><div>Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!<br></br><br></br><cite>Melanie Sunny said:…</cite></div>
</blockquote>
MAY GOD BLESS.<br />
<br />
<cite>cheryl cliff said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?id=1187424%3ATopic%3A85920&amp;page=2#1187424Comment87432"><div>Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!<br/><br/><cite>Melanie Sunny said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A87409&amp;xg_source=msg_com_forum#1187424Comment87409"><div>I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.<br/><br/>******************************************************************************************************************<br/><br/>Thank you for your message.<br/><br/>We plan a first clinical trial in patients..<br/><br/>Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.<br/>If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.<br/>Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.<br/><br/>With best wishes,<br/><br/><br/><br/><br/>Giulio Cossu<br/><br/>Division of Regenerative Medicine<br/>San Raffaele Scientific Institute<br/>Via Olgettina 58<br/>20132 Milan<br/>Tel: +3902 2643 4954<br/>Fax: +3902 2643 4621<br/>E-mail: cossu.giulio@hsr.it<br/><br/>Department of Biology<br/>University of Milan<br/>Via Celoria 26<br/>20129 Milan<br/>Tel: +3902 503 14798<br/>Fax: +3902 503 14781<br/>E-mail: giulio.cossu@unimi.it</div>
</blockquote>
</div>
</blockquote> Very Good! Thanks Melanie for…tag:community.parentprojectmd.org,2010-07-07:1187424:Comment:874322010-07-07T14:35:29.690Zcheryl cliffhttp://community.parentprojectmd.org/profile/cherylcliff
Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!<br />
<br />
<cite>Melanie Sunny said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A87409&amp;xg_source=msg_com_forum#1187424Comment87409"><div>I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr.…</div>
</blockquote>
Very Good! Thanks Melanie for contacting Dr Cossu and sharing what he said!!!<br />
<br />
<cite>Melanie Sunny said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A87409&amp;xg_source=msg_com_forum#1187424Comment87409"><div>I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.<br/><br/>******************************************************************************************************************<br/><br/>Thank you for your message.<br/><br/>We plan a first clinical trial in patients..<br/><br/>Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.<br/>If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.<br/>Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.<br/><br/>With best wishes,<br/><br/><br/><br/><br/>Giulio Cossu<br/><br/>Division of Regenerative Medicine<br/>San Raffaele Scientific Institute<br/>Via Olgettina 58<br/>20132 Milan<br/>Tel: +3902 2643 4954<br/>Fax: +3902 2643 4621<br/>E-mail: cossu.giulio@hsr.it<br/><br/>Department of Biology<br/>University of Milan<br/>Via Celoria 26<br/>20129 Milan<br/>Tel: +3902 503 14798<br/>Fax: +3902 503 14781<br/>E-mail: giulio.cossu@unimi.it</div>
</blockquote> Yes, I spoke with Debra Mille…tag:community.parentprojectmd.org,2010-07-07:1187424:Comment:874272010-07-07T14:28:02.834Zcheryl cliffhttp://community.parentprojectmd.org/profile/cherylcliff
Yes, I spoke with Debra Miller, CureDuchenne, who is helping with funding of Cossu's trial. She mentioned he wanted to start previously but is having trouble with the Italian regulatories. :( more delays but they do plan (perhaps hope) to begin 2011 like Raktim said.<br />
<br />
<cite>RAKTIM SINGH said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A87393&amp;xg_source=msg_com_forum#1187424Comment87393"><div>Hi…</div>
</blockquote>
Yes, I spoke with Debra Miller, CureDuchenne, who is helping with funding of Cossu's trial. She mentioned he wanted to start previously but is having trouble with the Italian regulatories. :( more delays but they do plan (perhaps hope) to begin 2011 like Raktim said.<br />
<br />
<cite>RAKTIM SINGH said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?commentId=1187424%3AComment%3A87393&amp;xg_source=msg_com_forum#1187424Comment87393"><div>Hi Rahul,<br/><br/>Prof Giulio Cossu from 'Division of Regenerative Medicine, San Raffaele Scientific Institute' is working on this. He is planning to use mesoangioblasts stem cells. Also, he had recruited DMD kids where full an HLA-matched donor (a brother or a non carrier sister) were found. I understand that he planning to start the trial in March 2011.<br/><br/>Raktim</div>
</blockquote> I sent an email to Dr. Giulio…tag:community.parentprojectmd.org,2010-07-07:1187424:Comment:874092010-07-07T13:46:24.499ZMelanie Sunnyhttp://community.parentprojectmd.org/profile/FengFeng
I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.<br />
<br />
******************************************************************************************************************<br />
<br />
Thank you for your message.<br />
<br />
We plan a first clinical trial in patients..<br />
<br />
Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most…
I sent an email to Dr. Giulio Cossu yesterday and this is what I received this morning from Dr. Cossu.<br />
<br />
******************************************************************************************************************<br />
<br />
Thank you for your message.<br />
<br />
We plan a first clinical trial in patients..<br />
<br />
Given the risk of an unprecedented trial in humans, we have selected initially patients with most severe Duchenne Muscular Dystrophy (DMD), age 6-9, still in good clinical conditions and, most importantly, with an HLA-matched donor (a brother or a non carrier sister). Recruitment is now closed. We have started a preliminary trial in 30 DMD children (with same eligibility criteria) to validate outcome measures in order to have sensitive and reproducible monitoring of possible force changes that may follow cell transplantation. Among these 30 patients we will will recruit three patients who will undergo cell transplantation, hopefully early (March) in 2011 and will know how it works in the end of the same year.<br />
If things work out well on this project, by 2012 we may plan new trials on older patients and/or affected by other forms of muscular dystrophy. The number of patients that we will be able to treat will be small because of logistic and economic problems. Other centers are learning this technique and, should things go well, may begin there also. But this takes a lot of time. Also do not forget that this is a hope. We have no guarantee that it will work.<br />
Meanwhile other trials have started and enroll larger number of patients. You may inquire about them at MDA or Duchenne Parent Project.<br />
<br />
With best wishes,<br />
<br />
<br />
<br />
<br />
Giulio Cossu<br />
<br />
Division of Regenerative Medicine<br />
San Raffaele Scientific Institute<br />
Via Olgettina 58<br />
20132 Milan<br />
Tel: +3902 2643 4954<br />
Fax: +3902 2643 4621<br />
E-mail: cossu.giulio@hsr.it<br />
<br />
Department of Biology<br />
University of Milan<br />
Via Celoria 26<br />
20129 Milan<br />
Tel: +3902 503 14798<br />
Fax: +3902 503 14781<br />
E-mail: giulio.cossu@unimi.it Hi Rahul,
Prof Giulio Cossu…tag:community.parentprojectmd.org,2010-07-07:1187424:Comment:873932010-07-07T07:53:21.366ZRAKTIM SINGHhttp://community.parentprojectmd.org/profile/RAKTIMSINGH
Hi Rahul,<br />
<br />
Prof Giulio Cossu from 'Division of Regenerative Medicine, San Raffaele Scientific Institute' is working on this. He is planning to use mesoangioblasts stem cells. Also, he had recruited DMD kids where full an HLA-matched donor (a brother or a non carrier sister) were found. I understand that he planning to start the trial in March 2011.<br />
<br />
Raktim
Hi Rahul,<br />
<br />
Prof Giulio Cossu from 'Division of Regenerative Medicine, San Raffaele Scientific Institute' is working on this. He is planning to use mesoangioblasts stem cells. Also, he had recruited DMD kids where full an HLA-matched donor (a brother or a non carrier sister) were found. I understand that he planning to start the trial in March 2011.<br />
<br />
Raktim Hi Rahul,
Sorry in the delay…tag:community.parentprojectmd.org,2010-07-04:1187424:Comment:869712010-07-04T16:47:10.996Zcheryl cliffhttp://community.parentprojectmd.org/profile/cherylcliff
Hi Rahul,<br />
Sorry in the delay with getting back to your question, we were away on holiday for a bit. Look for information related to stem cell work being done at the STEM CELL RESEARCH INSTITUTE, HSR, MILAN, ITALY. Also check into MESOANGIOBLASTS, and DR. G. COSSU. Don't hesitate to share what you find, OK?<br />
best<br />
cheryl<br />
<br />
<cite>Rahul Grover said:…</cite>
Hi Rahul,<br />
Sorry in the delay with getting back to your question, we were away on holiday for a bit. Look for information related to stem cell work being done at the STEM CELL RESEARCH INSTITUTE, HSR, MILAN, ITALY. Also check into MESOANGIOBLASTS, and DR. G. COSSU. Don't hesitate to share what you find, OK?<br />
best<br />
cheryl<br />
<br />
<cite>Rahul Grover said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?page=1&amp;commentId=1187424%3AComment%3A86715&amp;x=1#1187424Comment86715"><div>Hi Cheryl.<br/><br/>Can you please point me to where I can get more information on the stem cell trials in Italy. I haven't been able to find it.<br/><br/>many thanks!<br/>-Rahul<br/><br/><cite>cheryl cliff said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1#1187424Comment86394"><div>I suspect the scientists at GSK/Prosensa can't answer that question Katerina. The reason is because the answer you seek isn't up to them, it is up to the regulatory agencies at each country...FDA,EMEA, and the like. This is the first time we are faced with personalized medicine. It might be difficult to get the regulatory agencies to comprehend the need for this. On the other hand, there might be sympathetic people on these regulatory boards who want personalized meds to come through. It's a crap shoot either way. But I do believe in the survival instincts of the human race, and humans are on these regulatory boards. Plus, we've got Pat on our side. She is doing everything possible to make regulatories understand what we need. Gotta give her credit for all the hard work she's done.<br/><br/>As far as having hope...there is some, for your son too, more than EVER in the history of this disease. Exon skipping is moving along but there are other solid approaches too. And they aren't mutation specific. Look into Utrophin Upregulation, there is a phase I trial underway in the UK, Myostatin Inhibitors, with a phase II trial underway in Canada. And of course a phase I using Stem Cells going on in Italy.<br/><br/>Its hard to keep watching this stuff spin in the dmd universe and not have it ready for our boys now. Something will break. We have to remain hopeful.<br/><br/><br/><br/><cite>Katerina said:</cite><blockquote cite="http://duchennecommunity.ning.com/forum/topics/prosensa-and-glaxosmithkline-1?page=1&amp;commentId=1187424%3AComment%3A86367&amp;x=1#1187424Comment86367"><div>Is it possible to ask this question to the scientist in Prosensa. If these trials are successful in tolerability and outcome, will other exons still have to go through similar trials? I Will they eventually be able to say that the concept and the process is the same, just tailored individually, and be able to make exon skipping drugs for whichever exon they want skipped<br/>It's so hard live without hope, my son has the deletion of 21 exon and he need skipping of 20 or 22 exon....is there any chance...</div>
</blockquote>
</div>
</blockquote>
</div>
</blockquote>